USA/Canada (Toll-Free): +1-800-792-5285, +1-503-894-6022. SRP-9001 aims to treat DMD by delivering a gene that codes for a functional copy of dystrophin to the muscle tissues. Whole-body systemic gene therapy is likely the most effective way to reduce greatly the disease burden of Duchenne muscular dystrophy (DMD), an X-linked inherited muscle disease that leads to premature death in early adulthood. RGX-202 is intended to deliver a transgene encoding a novel microdystrophin with functional elements of the C-Terminal (CT) domain found in naturally occurring dystrophin. Duchenne Muscular Dystrophy life expectancy is between the ages of 16 and early 20s. Has developed a patented, high-performance cell-engineering platform for biopharmaceutical partners. Founded in 1998, uniQure is a Dutch biotech company that develops gene therapies for a variety of diseases, including hemophilia, Huntington's disease, and congestive heart failure. GALGT2 (Nationwide Childrens) is under clinical development by Sarepta Therapeutics and currently in Phase II for Duchenne Muscular Dystrophy. SRP-9001 aims to treat DMD by delivering a gene that codes for a functional copy of dystrophin to the muscle tissues. Participants in Part 2 of Study SRP-9001-102 scored 2.0 points higher on the mean North Star Ambulatory Assessment (NSAA) 48 weeks after SRP-9001 treatment compared to a pre-specified matched external control cohort (p value=0.0009). The companys platform is based on its pioneering work with phosphorodiamidate morpholino oligomer (PMO) chemistries. AAV RP-A501, LV RP-L102, LV RP-L201, LV RP-L301. EMBARK is currently recruiting males with DMD aged 4 to 7 in various locations across the United States. But we were cautious after the high profile death of Jesse Gelsinger in 1999.. Five years ago, scientist He Jiankui shocked his peers and the world with claims that he created the first genetically edited babies. In late 2019, Astellas Pharma Inc. (TSE: 4503) agreed to acquire Audentes Therapeutics for approximately $3 billion. Children with DMD tend to get stronger between 3 to 7 years old, then start to decline, Hesterlee explained. Importantly, our platform is Not Restricted By Gene Size. In May 2022, four companies, Pfizer, Sarepta, Genethon and Solid Biosciences, were all observing serious side effects in their gene therapy clinical trials for DMD. https://www.pharmalive.com/wp-content/uploads/2021/08/Mega-3-Billion-Deal-Shapes-Up-for-Roche-to-Target-AD-and-Parkinsons-BioSpace-8-24-21.jpeg, https://www.pharmalive.com/wp-content/uploads/2020/01/Pharmalive_4c-300x37.png, FDA accepts BLA for Roche-Sarepta's DMD gene therapy, Copyright - PharmaLive and Outcomes LLC |, Axsome headed to FDA after Phase III Alzheimers agitation win, Social Determinants of Health (SDOH): Three Trends to Watch in 2023, U.S. Centers for Disease Control and Prevention (CDC). Waiting in the wings is Pfizer, whose DMD hopeful PF-06939926encountereda roadblock late last year after a treated patient died. WebGene therapy Cell therapy Drug therapy Mutation specific approaches About clinical research Current trials in DMD Current trials in SMA Current trials in LGMD Facing the Challenges of Clinical Trials Overview of therapeutic approaches for SMA The Problem The splicing process Therapeutic strategies for SMA Outcome measures It could convert this disease from a devastating diagnosis to a manageable disease in the next 10 years.. Duchenne muscular dystrophy (DMD) is a severe genetic disorder characterized by progressive muscle degeneration and consequent muscle weakness. Vyondys 53 (golodirsen) Injection. WebDuchenne Muscular Dystrophy (DMD) Core Dataset; Facioscapulohumeral Muscular Dystrophy (FSHD) Core Dataset DMD Research overview. This designation is designed to provide regulatory assistance and financial benefits to the therapys clinical research and evaluation, as well as a seven-year period of marketing exclusivity in the United States after regulatory clearance. What about a tourniquet and pressure? AAV-RPGR, AAV-RPE65, AAV-CNGB3, AAV-CNGA3, AAV-AIPL, A007, A008, A006, AAV-CNGB3, AAV-CNGA3, AAV-AIPL, A007, A008, A006. Dystrophin, Byrne says, is the largest protein-coding gene in the body and does not fit in an AAV vector. Both employ exon skipping, redirecting DNA processing inside the muscle cells to create minidystrophin right in the cells much like the researchers did in the lab, but directly in the children themselves. In recent years, weve gotten much better at detecting benefits in the boys even when they are in the early stages and improving, so trials have started to skew younger, including children as young as 4 years old.. The Agency has also granted the therapy priority review and set the regulatory action date for May 29, 2023. They are currently developing gene therapies using CRISPR/Cas9 technology. The biotech aims to engineer precision genetic medicine for rare diseases. The first signs of DMD appear as the young boys begin to walk and get more mobile, typically between the ages of 2 to 5. WebThere are many challenges facing the management of DMD patients in the United Arab Emirates and Kuwait and most likely other countries within the Middle East. HuCo kidney, HuCo heart, HuCo islet cells, HuCo liver perfusion. Life-threatening severe DMD complications may eventually develop, such as cardiomyopathy and respiratory difficulties. By Chelsea Weidman Burke. This news closes a tumultuous time for Astellas regarding the therapy. Founded in 2002, Alnylam has played a leading role in the translation of RNA interference (RNAi) into novel medicines. Currently these trials are taking place in the US. The earlier you treat, the better, but its hard to measure benefit if the children are not yet manifesting a lot of symptoms, so you want to test the children at a stage when theyre progressing, said Hesterlee. It is developed based on exon skipping technology. He is currently a Professor of Physiology and Biophysics at the University of Washington. First, the good news for Solid Biosciences: the new process put in place for its Duchenne muscular dystrophy gene therapy project SGT-001 looks safe. The Phase, I/II trial, named AFFINITY DUCHENNE study, which is set to begin in the coming months. Four of those are for ocular indications while the other two are for a salivary gland condition and Parkinsons disease. The company then opened U.S. enrollment for a Phase III trial of the therapy that was already underway in the U.K., Canada and other countries. PF-06939926was granted Fast Track designation in 2020. The Several gene therapy approaches are being explored as treatments for Duchenne muscular dystrophy (DMD). SRP-9001 is a gene therapy candidate for Duchenne Muscular Dystrophy treatment. In addition, Brian covered the medical device sector for 10 years at UBM. The company is using a stem cell biology and genomics platform to develop a novel autologous induced pluripotent stem cell (iPSC)-derived neuron replacement therapy for Parkinsons Disease. Its commercial products include Exondys 51, Vyondys 53 and Amondys 45 indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene. "Within the context of Duchenne and other rare diseases, it's a very robust sample size and one that will grow with data from EMBARK.". Eventually, they will need ventilation to help them breathe. Duchenne Muscular Dystrophy (DMD) is an X-linked disease that is inherited. The company aims to create novel non-viral genetic medicine that supports long-term efficacy while providing support for redosing, if needed. In November 2021, RGX-202 was designated as an orphan drug by the FDA for Duchenne Muscular Dystrophy treatment. It is a recombinant adeno-associated virus serotype 9 (AAV9) capsid containing a shortened version of the human dystrophin gene (mini-dystrophin) controlled by a human muscle specific promotor. Microdystrophin expression was seen via muscle biopsies 90 days after treatment (at a dose of 2E14 vg/kg), which stabilized dystrophin-associated proteins and restored activity of a key enzyme (called neuronal nitric oxide synthase, or nNOS) in the muscles. Its important to realize that the major goal of an animal study is not necessarily to show efficacy, he said. We know whats wrong, well fix it! Hesterlee added. The factor that is expected to restrain the growth of market is the huge price tag associated with drugs. Sarepta Therapeutics said topline results from Part 2 of its study SRP-9001-102, an ongoing, randomized, double-blind, placebo-controlled clinical trial to evaluate the safety, efficacy and tolerability of a single dose of its gene therapy for the progressive neuromuscular condition Duchenne muscular dystrophy, showed statistically It is currently being investigated in a Phase I/II study in six boys ages 4 and up. Rare Daily Staff. It is usually observed between the ages of three and six. SGT-001 has received Rare Pediatric Disease and Fast Track Designation in the United States and Orphan Drug Designation in the US and EU in 2017. At 12 months post-injection, the boys had sustained, significant improvement in minidystrophin expression and improved muscle function (measured via the NSAA rating scale). Operations, Competitive Intelligence, Competitive Landscaping, and Mergers & Acquisitions. Three serious adverse events (SAEs) occurred, but they fully resolved within two weeks. In May, Pfizer, Sarepta, Solid and Genethonjoined armsto investigate why they were all being tripped up by serious safety concerns. When expanded it provides a list of search options that will switch the search inputs to match the current selection. This would appear to be an easy solution. Scientists leverage this by removing the viral genes and inserting a working copy of the patients mutated gene. Instead of delivering the dystrophin gene, GALGT2 delivers the GALGT2 gene, which is also important for muscle function. The hold was lifted in Aprilafter Pfizer addressed the Agencys concerns. Patients with this form of the muscle-wasting disease don't make enough dystrophin, a protein SRP-9001 was safe and well-tolerated up to one-year post-administration. It employs a non-lethal modified virus (AAVrh74) with a high affinity for muscle tissue, allowing for targeted delivery. Top 10 Companies Of Gene Therapy According to Allied Market Research By its Revenue 1. Once inside the cell, the viral vector behaves like a virus and makes the cell produce the protein encoded by the working gene it is carrying, compensating for the original mutated copy. All rights reserved. According to data from Solids clinical program, SGT-001 has the potential to slow or stop the Duchenne progression, regardless of genetic mutation or disease stage. The clinical-stage biopharmaceutical company is focused on developing therapies for cancer and other immune-related diseases. Dystrophin, a protein present on the inner side of the membranes of skeletal and cardiac muscle cells, is controlled by the DMD gene. Arising in one of every 3,500 to 5,000 male infants worldwide, DMD is a rare neuromuscular disease caused by mutations in the gene encoding for the protein dystrophin. There are currently four companies who have DMD gene therapy products that have been given to boys with Duchenne, and three of the companies have ongoing studies in the USA. Their first gene therapy product, Luxturna, was approved by the FDA in 2017 to treat a form of inherited blindness. NTLA-2001, NTLA-2002, NTLA-2003, NTLA-3001, OTQ923/HIX763, NTLA-5001, NTLA-6001. As per DelveInsight Duchenne Muscular Dystrophy Epidemiology Report, the total DMD prevalent population was more than 30K in the 7MM in 2020, which is further expected to increase by 2032. Before coming to WTWH, he served as content director focused on connected devices at Informa. Reference: Barry Byrne, Joe Kornegay, et al., Assessment of systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophy, Science Translational Medicine (2023), DOI: 10.1126/scitranslmed.abo1815, Feature image: The protein dystrophin. They are currently developing gene therapies for a range of diseases, including Duchenne muscular dystrophy and hemophilia. WebDespite scientific discoveries in the field of gene and cell therapy, some diseases still have no effective treatment. AccordingAccording to Solid's leadership, this this would allow the company to focus on two key programs that hold the highest potential for DMD. Genetically, DMD is due to null mutation of the dystrophin gene, one of the largest genes in the genome. Founded in 2013, Editas Medicine is a biotech company based in Cambridge, Massachusetts that focuses on developing gene therapies using CRISPR/Cas9 technology. Medical Design and Outsourcing. Cell and gene therapies promise to enable broad changes in the healthcare system over the next decade, prompting a growing number of cell and gene therapy companies to join the space. areas Published: Jul 29, 2020 The drug in question, GS-1811 (formerl AbbVie Secures Fourth FDA Approval for Vraylar AbbVie has received its fourth FDA approval for Vraylar, adding major depressive disorder (MDD) adjunctive therapy to a list that includes schizophrenia and manic and depressive episodes in bipolar disorder. Solids is different because it contains the binding spot for an enzyme called nitric oxide synthase both Sarepta and Pfizer cut that portion out.. GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article. The only Duchenne Muscular Dystrophy treatment available are steroids like dexamethasone and gene-targeting therapies including exon skipping from Sarepta Therapeutics and NS Pharma for two small subsets of patients. The company boasts that its AI Workbench can help manage the complexity in RNA biology while identifying novel targets. Can FcRn Antagonists Be The Game-Changer in the Generalized Myasthenia Gravis (gMG) Treatment Market? At Qmed, he overhauled the brands news coverage and helped to grow the sites traffic volume dramatically. REGENXBIO (RGNX) is developing a gene therapy candidate, RGX-202, for treating DMD, which is currently in the pre-clinical stage. Louise Rodino-Klapac, CSO, executive VP and head of R&D, Sarepta Permission granted by Sarepta If approved, SRP-9001, would be the first gene therapy for the muscular degenerative disease known as DMD and is slated for complete evaluation under the accelerated approval path by the end of May 2023. Importantly, there were no serious adverse events (only mild to moderate events). Somatic gene therapy involves modifying genes in non-reproductive cells, such as cells in the skin or blood. Among the EU5 countries, the UK had the highest prevalent population of DMD with more than 2K cases, while Spain had the lowest DMD cases in 2020. With funding from biotech companies and the US Department of Defense, a blinded, placebo control study in dogs was approved. All rights reserved. Sarepta Therapeutics said topline results from Part 2 of its study SRP-9001-102, an ongoing, randomized, double-blind, placebo-controlled clinical trial to evaluate the safety, efficacy and tolerability of a single dose of its gene therapy for the progressive neuromuscular condition Duchenne muscular dystrophy, showed statistically A Non-Viral delivery method is much less likely to elicit an immune response, enabling repeated dosing over months or years. Pfizers PF-06939926 is an investigational gene therapy for Duchenne Muscular Dystrophy treatment. The biotech specializes in creating gene therapies for severe genetic disorders and cancer. GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. The FDA has accepted Roche and Sareptas Biologic License Application for the accelerated approval of SRP-9001 (delandistrogene moxeparvovec), an Other hurdles of developing a DMD gene therapy. Both Sarepta and Pfizer have collected some promising functional data, commented Hesterlee. Another challenge hinges on the fact that the gene is delivered using a virus, making the gene therapy an immunization in a way. The European Commission (EC) has granted orphan drug designation to AB-1003, an investigational gene therapy for limb-girdle muscular dystrophy type 2I/R9 (LGMD 2I/R9), being developed by Asklepios BioPharmaceutical (AskBio). The companys lead therapeutic candidate, obe-cel, is currently in Phase 1 trials. Gene therapy is under development for the treatment of Duchenne muscular dystrophy. Sarepta and Pfizer are evaluating their lead candidates for gene therapy in the late stages. They can help slow down disease progression, but tackling inflammation only addresses one downstream effect.. Companies focusing on DMD gene therapies have proceeded cautiously after a fatal case of myocarditis was observed in Pfizers gene therapy candidate. It is very likely that one or both of these gene therapies could be approved., This opens up the door for combination therapies, such as gene therapies to stabilize the muscle and small molecule drugs to deal with downstream events like fibrosis and inflammation, Hesterlee concluded. The drug is also known as rAAVrh74.MHCK7.micro-dystrophin due to its construction. The findings showed that the microdystrophin protein remains expressed and functional in biopsy samples collected 12 to 24 months after SGT-001 administration. The collaboration could be worth more than $3 billion. Five pharmaceutical companies, namely Sarepta Therapeutics, Roche, Pfizer, Solid Biosciences, and Regenxbio, are currently working on gene therapy for Duchenne Muscular Dystrophy. However, for Duchenne muscular dystrophy gene therapies, the size of the dystrophin gene is a problem. They also saw a decrease in the loss of skeletal muscle function in those who received treatment versus those who didnt. AAV has a limited gene size capacity of 5 kilobases (kb), precluding its use for many larger genes. In mid-June, Sarepta announced that preliminary results from four boys ages 4-7 years were published in JAMA Neurology. Viruses are very well evolved to get into cells, commented Hesterlee. The company has a variety of in vivo and cell therapy programs for indications including hemophilia A and various tumors. Increase in the prevalence of chronic disorders, rise in government support, and ethical acceptance of gene therapy for cancer treatment drive the growth of the global gene therapy market. The leading companies developing gene therapy candidates for DMD are Sarepta Therapeutics, Roche, Pfizer, Solid Biosciences, and Regenxbio. Sometimes called minidystrophins, there are slight variations between different versions of these shortened genes, but the key is they are all small enough to fit into AAV, explained Hesterlee. Their gene therapy product, Glybera, was the first gene therapy to be approved in Europe in 2012. They are currently focused on developing gene therapies for a range of diseases, including cancer and genetic disorders. This type of gene therapy is currently the most commonly used approach and has been successful in treating several genetic diseases. Next, the bad: interim data from the phase I/II Ignite DMD trial are disappointing, and the groups stock slid 24% this morning. In May 2022, four companies, Pfizer, Sarepta, Genethon and Solid Biosciences, were all observing serious side effects in their gene therapy clinical trials for DMD. We discovered in 1997 that AAV vectors can be delivered to muscle cells and have a therapeutic benefit, said Byrne. Based in California, Audentes Therapeutics is a biotechnology company that employs gene therapy technology to develop treatments for people with rare muscle Specializing in CRISPR/Cas9 technology, CRISPR Therapeutics is initially targeting the blood diseases -thalassemia and sickle cell disease. WebThe Roche Groups bold commitment to gene therapy collaborations across the organisation and industry are a stake in the ground: the possibility and potential of using In January, The FDA approved Regenxbios request to conduct a Phase I/II clinical trial in the United States to assess the safety and efficacy of RGX-202, its experimental gene therapy for Duchenne Muscular Dystrophy (DMD). The companys Tapestri platform can simultaneously. 617). MHCK7 is intended to increase gene activity in the heart and skeletal muscles, which are the most affected muscle groups in DMD patients. Focuses on developing novel gene therapies for rare inherited genetic diseases. anti-BCMA CA, anti-CD20 CAR, anti-CD19, CD20 BICA, AFP TCR-T. 2020 by Myosana Therapeutics, Inc.. eGenesis has a pipeline of gene therapies focused on inherited, systemic, debilitating chronic diseases. DMD has a life expectancy of 16 to early 20s. They also have 12 other exon skipping-based genetic medicines in their pipeline. The clinical-stage regenerative medicine company specializes in using placental cells and proprietary, 3-D technology platform to develop cell therapies for inflammation, muscle injuries, hematological disorders and radiation exposure. How Healthcare Apps are Adding New Perspectives to the Healthcare Industry? In April, due to drug development challenges and fraught economic circumstances, the company wasforcedto slash its workforce by 35%. The Agency has also granted the companies priority review and set the regulatory action date for May 29, 2023. Sarepta and Rocheenteredinto a partnership in December 2019, with Roche surrendering $1.15 billion upfront for exclusive rights to SRP-9001. For example, the dystrophin gene is too large to fit into the adeno-associated viruses, or AAVs, that are commonly used to deliver gene therapies. That allowed researchers to test the gene therapy proof-of-concept in DMD patients without worrying about systemic administration right off the bat. Sarepta Therapeutics. Arrowhead Pharmaceuticals specializes in developing therapies to treat intractable diseases by silencing the genes responsible for them. The biopharma is developing genome-edited off-the-shelf CAR-T and CAR-NK cell therapies for various tumor types. An IND application is expected by the end of 2021. Regardless, Pfizer will need to examine the situation and acquire the data necessary to continue the Phase Ib trial and make changes to future trials, such as omitting certain mutation types. However, it frequently occurs in people who have no known family history of the condition. Email (801) 436-5597. Sarepta is responsible for SRP-9001's U.S. application. In 2019, it spent $4.3 billion to acquire gene therapy specialist Spark Therapeutics. The company is developing a pipeline of NAM-enabled cell therapies for a range of diseases with significant unmet medical need. ORLANDO, FloridaJeffrey Chamberlain, PhD, outlined the 4 different types of gene therapy for treating Duchenne muscular dystrophy (DMD) at the Gene Therapy and Gene Editing Symposium which took place on the second day of the CureDuchenne 2022 FUTURES National Conference . Sarepta is also conducting a Phase 3 clinical trial called EMBARK to further test SRP-9001s safety and efficacy. He has extensive experience in the Bio-pharmaceutical field, with positions at Pfizer, as Medical Director of Rare Diseases and, more recently Moderna, where he was responsible for taking the first mRNA therapeutics for rare diseases into the clinic. Byrne and colleagues now had a therapeutic that would fit in the AAV. Click for Index It has a diverse approach to cellular therapy using nicotinamide (NAM) to expand multiple cell types. DMD is the most frequent type of muscular dystrophy that develops in childhood and primarily affects men. Vertex has acquired Exonics and has a partnership with CRISPR Therapeutics to develop a gene-editing platform for Duchennes muscular dystrophy (DMD) and myotonic dystrophy (DM1). The companys pipeline includes programs focused on GM1 gangliosidosis, Krabbe disease and frontotemporal dementia. In patients with Duchenne muscular dystrophy, the affected gene codes for the protein, dystrophin, which acts as a shock absorber between muscle cells and connective tissue, as well as supporting muscle contraction. These findings showed a significant improvement in patient-reported outcomes and provided encouraging evidence of functional benefit 1.5 years after treatment when compared to natural history data. Moreover, a thorough clinical assessment, a complete patient history, and a number of specialist diagnostics, including molecular genetic tests, are used for Duchenne Muscular Dystrophy diagnosis. What is now called golden retriever muscular dystrophy (GRMD) turns out to be an excellent model for Duchenne muscular dystrophy because it involves the canine dystrophin gene, causes similar symptoms, and is also X-linked. The trials main purpose is to monitor changes in NSAA scores. Fixing the mutated gene (through gene editing) or providing cells with a new healthy copy of the gene (through gene therapy) would provide the best benefit, possibly even leading to a lifelong cure. WebDr Paul Benson is an oral and facial surgeon, serial entrepreneur and business coach with a diverse portfolio of companies in a variety of industries including healthcare, beauty, MedTech 100 is a financial index calculated using the BIG100 companies covered in Feb 18, 2022 | Reading Time: 8 minutes. These DMD therapies may, at best, slow the progression of Duchenne. Its lead candidate, CAP-1002, is an off-the-shelf cardiac cell therapy now in late-stage clinical development for Duchenne muscular dystrophy. It is difficult and costly to manufacture large quantities of AAV. Germline gene therapy, on the other hand, involves modifying genes in reproductive cells, such as eggs or sperm. SRP-9001 includes a different serotype of AAV, called AAVrh74 (which also gets into muscle and heart cells well), and a microdystrophin gene. The company develops its pipeline products using its multi-platform Precision Genetic Medicine Engine in gene therapy, RNA, and gene editing. Bayer created a cell and gene therapy platform in 2019 within its pharmaceutical division. 6 min read. Without dystrophin, the muscle cells suffer from microtears, leading to their demise and progressive muscle wasting. Antibody status can be quite divisive in the DMD community.. Duchenne UK and the DMD Hub wanted to understand what more can be done to encourage them to be omidubicel, GDA-20, GDA-301, GDA-401, GDA-501, GDA-601. Sareptas lead gene therapy candidate is SRP-9001, an AAV-mediated micro-dystrophin gene therapy, which is being evaluated in a phase I/II study for DMD.

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